FDA changes to compassionate use regs a new blow for Sarepta over eteplirsen

The US Food and Drug Administration's streamlining of the process for physicians to seek ‘compassionate use’ of investigational treatments for their patients could make it more unlikely that Sarepta Therapeutics (Nasdaq: SRPT) drug eteplirsen will gain accelerated approval.

That is the speculation that followed the statement released Thursday by FDA Commissioner Robert Califf.

“As a physician, I understand the importance of being able to access investigational treatments for a patient when there are no other options to treat their serious disease or condition,” his statement read, adding: “Access to investigational treatments requires the active cooperation of the FDA, industry, and health care professionals in order to be successful. But we know that navigating that process can be challenging and time consuming, and we are committed to reducing the procedural burdens on physicians and patients whenever possible.”